Advocates looking to support the development of new treatments for a rare disease can follow many different paths. But they are more likely to succeed in finding partners if they have the right tools in place.  The following resources will help you understand the processes involved in developing new therapies, from basic research to clinical trials, and beyond.

Global Genes is hosting the RARE Drug Development Symposium June 8-10, 2022 in Philadelphia, PA, bringing together rare disease advocacy leaders and researchers to identify barriers to research, envision solutions, and accelerate progress for our communities. 

Register to Attend 

Don’t miss the upcoming webinar, What Does it Take to Become a Research-Ready Organization with Travere Therapeutics on May 12, 2022  6:00pm ET where you’ll learn about the research process and how to make sure you have the right pieces in place to attract researchers and industry investors. This webinar will cover terms and concepts you need to know to prepare your community to engage with researchers and biotech, including:

  • The building blocks of basic and translational research and clinical trials
  • Role of advocates in gathering data, improving diagnosis and expanding awareness
  • Hows and whys of patient endpoint selection
  • Working with regulatory bodies
  • Getting up to speed on new treatments and innovative trial model

Register for Webinar

RESOURCES: Introduction to Drug Development 

Understanding Drug Development

A free, in-depth, Rare University course on drug development for advocates and families.

From Molecules to Medicine

This series of toolkits provide an overview of drug development, including:

Basic Tools of Research

Learn about the basic tools available to researchers, including cell-based models  (such as IPS cells and CRISPR), organoids, in vivo models therapeutic modalities, computational approaches (AI)

 

Drug Development Road Map

High-level, infographic approach to the topic with a helpful glossary of terms

Drug Development White Paper

Describes the must-have collaborations for drug development

Partnering with Researchers and Industry

Starting a Research Program from the Ground Up

The story of a caregiver who began a relentless search for treatment when her two-year-old- daughter hemorrhaged to shock in her crib from an undiagnosed bowel disease.

How to be a Great Research Partner

This panel from the 2020 Rare Drug Development Symposium focuses on how advocacy groups can be involved at every step and what makes industry and researchers trusted partners.

Building a Toolbox for Research

Covers the business of science, how to fund academics, and putting together the pieces of a research program 

Mapping Patient Inclusion in Drug Development

 A discussion of the lifecycle of traditional drug development and new trends in research, as well as how to partner with academia and industry.

Data DIY 3

How to develop a research network and convene experts to set a research strategy.

How Patients Can Share Their Voices in the Drug Development Process

Learn how the participation of those living with a rare disease can help researchers and regulators design research protocols and identify meaningful endpoints.

Registries and Natural History Studies

Data DIY 1 

Explores the whys and hows of data collection 

Data DIY 2

Focuses on data trusts, governance and collection platforms

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